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Four decades of stem cell transplantation for Fanconi anaemia in the Netherlands
Author(s) -
Smetsers Stephanie E.,
Smiers Frans J.,
Bresters Dorine,
Sonnevelt Martine C.,
Bierings Marc B.
Publication year - 2016
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.14165
Subject(s) - medicine , busulfan , transplantation , hazard ratio , fludarabine , cohort , cyclophosphamide , hematopoietic stem cell transplantation , regimen , stem cell , surgery , total body irradiation , gastroenterology , oncology , chemotherapy , biology , confidence interval , genetics
Summary This article presents the haematopoietic stem cell transplantation ( SCT ) results of the complete Dutch Fanconi anaemia ( FA ) patient cohort. Sixty‐eight Dutch FA patients have been transplanted since 1972. In total, 63 (93%) patients engrafted, 54 after first SCT and 9 after second SCT . Fludarabine (FLU)‐based conditioning was associated with decreased graft failure (odds ratio 0·21, P = 0·01), decreased early mortality (hazard ratio 0·25, P = 0·01) and improved 5‐year overall survival (FLU 87·8% [standard error ( SE ) 5·1%] versus non‐FLU 59·3% [ SE 9·5%], P = 0·01). Late mortality was mainly caused by squamous cell carcinoma. Twenty‐two patients were treated with the current Dutch FA conditioning regimen (FLU 150 mg/m 2 and cyclophosphamide 30 mg/kg ± anti‐thymocyte globulin ‐ no irradiation). Stem cell donors were matched related (n = 8) or alternative donors ( n = 14). Stable engraftment after first SCT was achieved in 19 (86%) patients. At a median follow‐up of 3·9 years 20 (91%) patients are alive. Our study provides a unique overview of a nation‐wide SCT cohort illustrating the major improvements in treatment regimen and patient outcome in recent years. It shows that a non‐irradiation and busulfan‐free conditioning regimen can be used successfully, also in alternative donor SCT . Furthermore, it underlines the importance of late cancer screening and comprehensive care for this complex disorder.